California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia

Gen 13, 2026. The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility. The new funding will help the research team complete the final steps required by federal regulators before they can apply to begin a first-in-human clinical trial.

Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to SGT-212 for the treatment of Friedreich’s ataxia (FA). Additionally, earlier today, the Company reported that the first participant has been dosed in FALCON, a Phase 1b, first-in-human clinical trial evaluating SGT-212 for the treatment of FA.